Drug Discovery World

DECEMBER 21, 2023

In a record year for gene therapy approvals, it isn’t surprising that cell & gene therapy (CGT) has been a hot topic. Other biotechnologies coming to the fore this year were antibody-drug conjugates and CRISPR gene editing. DDW Multimedia Editor Megan Thomas explored three trends in the market.

Gene Therapy 59

Gene Therapy Gene Antibody FDA Approval 59

XTalks

OCTOBER 20, 2023

Clinical-stage genome editing company Intellia Therapeutics has received clearance from the US Food and Drug Administration (FDA) for its Investigational New Drug (IND) application to start a pivotal phase III trial of NTLA-2001 for the treatment of transthyretin (ATTR) amyloidosis with cardiomyopathy.

In-Vivo 52

In-Vivo Gene Editing Trials Gene 52

Drug Discovery World

MAY 6, 2024

During the period 2016-2021, the Brazilian, Chinese and Indian markets grew by 11.7%, 6.7% respectively compared to an average market growth of 5.8% for the top five European Union markets and 5.6% for the US market 2. However, Europe is facing increasing competition from emerging economies.

Drugs 59

Drugs Life Science Gene Editing Gene 59

Pharma Marketing Network

DECEMBER 21, 2020

Almost two decades after the human genome was sequenced, a trickle of new genetic medicines (i.e., those that modify the expression of an individual’s genes or repair abnormal genes) has entered clinical practice, including 11 RNA therapeutics, 2 in vivo gene therapies, and 2 gene-modified cell therapies.

Genetics 52

Genetics Medicine Gene Therapy Marketing 52

Drug Discovery World

JULY 10, 2023

If there’s one thing I would take from the Summer 2023 issue of Drug Discovery World , it would be that the application of technologies and processes throughout the pharmaceutical industry are changing how scientists pursue the development of therapies. There are myriad ways that this is being enabled.

Gene Therapy 52

Gene Therapy Gene Scientist Gene Editing 52

Drug Discovery World

OCTOBER 16, 2023

Dr Salako says there are some clear fields of work associated with different areas, which include: Hertfordshire as a cell and gene therapy hub, with a number of companies in this space clustered around Stevenage. billion in funding. This is reflected by our recent share price performance outperforming the index in the past year.”

Drugs 75

Drugs Life Science Gene Therapy Genome 75

pharmaphorum

AUGUST 31, 2022

After a recent approval, there are now three gene therapies available on the US market. Though there are still few commercial treatments, in terms of therapeutic potential, Ben Hargreaves finds that companies and investment firms are pushing for this number, and the overall market, to grow rapidly in the coming years.

Gene Therapy 98

Gene Therapy Gene Gene Editing DNA 98

Cloudbyz

MARCH 6, 2024

Additionally, there is a growing need for regulatory frameworks to ensure the safety, efficacy, and affordability of biologic therapies, particularly as they become more prevalent in clinical practice. Looking ahead, the future of pharmaceutical therapies is undeniably intertwined with the continued advancement of biologics.

Gene 75

Gene Immune Response Manufacturing Gene Therapy 75

Pharmaceutical Technology

FEBRUARY 13, 2023

Several biotech companies and researchers are now exploring medical devices and gene therapies to address not just common forms of epilepsy, but also rare conditions such as Dravet Syndrome. Common gene therapies use vehicles such as adeno-associated vectors (AAVs), which cannot carry the required payload in this case, says Carney.

Research 264

Research Gene Gene Therapy Genome 264

The Pharma Data

NOVEMBER 3, 2020

LogicBio’s proprietary genome editing technology platform, GeneRide, enables the site-specific integration of a therapeutic transgene without nucleases or exogenous promoters by harnessing the native process of homologous recombination.

Clinical Trials 52

Clinical Trials Clinical Trials Gene Editing Genome 52

XTalks

DECEMBER 20, 2023

CRISPR Gene Editing Therapeutics CRISPR/Cas-9 technology makes lists like this almost every year, and for good reason. Given its ability to precisely edit genes, there is tremendous promise for the revolutionary technology to offer cures for genetic disorders and to treat various diseases.

Life Science 119

Life Science Gene Editing Development Clinical Trials 119

Drug Discovery World

JANUARY 26, 2023

DDW Editor Reece Armstrong takes a look at the pharmaceutical research and development market in 2023 and asks key opinion leaders what challenges and trends will emerge throughout the year. billion showed the pharma giant taking further steps into the oncology market and developing its speciality medicines vaccines portfolio.

Development 98

Development Drugs Clinical Trials Clinical Trials 98

XTalks

FEBRUARY 27, 2024

Technologies like CRISPR-Cas9 have revolutionized the potential for gene editing, offering hope for curative treatments for conditions like Duchenne muscular dystrophy (DMD) and certain types of inherited blindness. Both therapies were awarded their approvals on the same day in December 2023.

Research 59

Research Drugs Gene Therapy Clinical Trials 59

Drug Discovery World

MAY 30, 2023

Erin Goodhue, Director of Clinical Research and Development, and Dawn Faller, Senior Manager, Global Clinical Marketing, Therapeutic Systems, Terumo Blood and Cell Technologies: “When thinking about cancer, breast cancer has surpassed lung cancer as the most diagnosed cancer worldwide with one in eight women diagnosed in their lifetime.

Research 52

Research Drugs Protein RNA 52

Pharma Marketing Network

JANUARY 20, 2021

Regarding the use of direct-acting anti-SARS-CoV-2 therapeutics (such as nucleoside analogs, protease inhibitors, and monoclonal antibodies), the panel stressed the need for constant surveillance to track changes in the viral genome that might result from their use.

Vaccination 52

Vaccination Vaccine In-Vivo Genetics 52

XTalks

DECEMBER 24, 2020

From isolating SARS-CoV-2 in early January to sequencing its genome shortly thereafter and having a prototype vaccine against it within days, scientific process and progress have held steadfast throughout the pandemic. CRISPR Gene Editing Inventors Win Nobel Prize. The illness was later named coronavirus disease 2019 (COVID-19).

Life Science 111

Life Science Vaccination Vaccine Gene 111

Pharmaceutical Technology

FEBRUARY 20, 2023

This may lead to therapies and dosing regimes being less suitable for minority populations – and according to Hans Bunschoten, chief strategy officer at clinical research company Cerba Research, part of Cerba Healthcare, this is a major problem. The technology platforms are almost the same.

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Research Clinical Trials Clinical Trials Trials 130